CAMBRIDGE, Mass.--(BUSINESS WIRE)--
Syros Pharmaceuticals (NASDAQ: SYRS) announced today that the first
patient has been dosed in the Phase 2 clinical trial of its lead drug
candidate, SY-1425, a first-in-class selective retinoic acid receptor
alpha (RARα) agonist, in genomically defined subsets of patients with
relapsed or refractory acute myeloid leukemia (AML) or high-risk
myelodysplastic syndrome (MDS) identified using a novel biomarker
discovered by its gene control platform.

“This is an important milestone for Syros and for patients,” said David
A. Roth, M.D., Chief Medical Officer of Syros. “There has been little
improvement in the treatment of AML and MDS for the past 20 years, and
survival rates for these patients lag behind many other blood cancers.
Treatment with SY-1425 represents a promising therapeutic strategy for
subsets of AML and MDS patients with a novel biomarker that we
discovered using our gene control platform. Our pioneering approach is
designed to advance a new wave of medicines to control the expression of
disease-driving genes in genomically defined subsets of patients to
provide them with a profound and durable clinical benefit. Our goal is
to rapidly advance this first-in-class therapy for these currently
underserved patients."

Using its gene control platform, Syros discovered subsets of AML and MDS
patients whose tumors have a highly specialized regulatory region of
non-coding DNA, known as a super-enhancer, that is associated with the RARA
gene, which codes for the RARα transcription factor. The super-enhancer
is believed to lead to over-production of the RARα transcription factor,
locking cells in an immature, undifferentiated and proliferative state.
Syros further investigated this unique biology directly in patient
tissues and conducted preclinical studies showing that the RARA
super-enhancer is predictive of response to treatment with SY-1425 in
preclinical models of AML. Based on that data, Syros is implementing a
biomarker strategy for its Phase 2 trial that selects a subset of
approximately 25 percent of AML and MDS patients who may respond to
treatment with SY-1425.

“The prognosis for these patients is poor, and targeted approaches like
SY-1425 offer hope for much-needed new therapies that attack the
underlying biology of the disease and hopefully allow patients to live
longer without the toxicities of traditional chemotherapy,” said Rachel
J. Cook, M.D., M.S., assistant professor of medicine at Oregon Health &
Science University and an investigator in the trial. “We’re pleased to
have enrolled the first patient in this clinical trial and look forward
to further investigating SY-1425 for this newly identified subset of AML
and MDS patients.”

The Phase 2 clinical trial is a multi-center, open-label trial exploring
safety and efficacy in relapsed or refractory AML or high-risk MDS
patients who have been prospectively selected using the Company's
biomarker strategy. The trial is expected to enroll approximately 40
patients, and the primary endpoint of the trial will be overall response
rate. The trial will also assess pharmacodynamic biomarkers, duration of
response, safety and tolerability, and survival. Additional details
about the trial can be found using the identifier NCT02807558 at www.clinicaltrials.gov.

SY-1425 is approved in Japan as Amnolake (tamibarotene) to treat a
different form of AML known as acute promyelocytic leukemia (APL), in
which it has a well-established efficacy and safety profile. Syros
in-licensed SY-1425 to develop and commercialize it in North America and
Europe for all cancers.

About Syros PharmaceuticalsSyros Pharmaceuticals is
pioneering the understanding of the non-coding region of the genome to
advance a new wave of medicines that control expression of
disease-driving genes. Syros has built a proprietary platform to
systematically and efficiently analyze this unexploited region of DNA in
human disease tissue to identify and drug novel targets linked to
genomically defined patient populations. Because gene expression is
fundamental to the function of all cells, the Company’s gene control
platform has broad potential to achieve profound and durable benefit
across a range of diseases. Syros is focused on cancer and
immune-mediated diseases and is advancing a growing pipeline, including
its lead drug candidates SY-1425, a selective RARα agonist for
genomically defined subsets of patients identified by its platform, for
a range of cancers including acute myeloid leukemia and myelodysplastic
syndrome, and SY-1365, a selective CDK7 inhibitor for a range of blood
cancers and solid tumors. Led by a team with deep experience in drug
discovery, development and commercialization, Syros is located
in Cambridge, Mass.

Cautionary Note Regarding Forward-Looking StatementsThis
press release contains forward-looking statements within the meaning of
The Private Securities Litigation Reform Act of 1995, including without
limitation statements by the Company regarding: the potential
therapeutic benefit of treatment with SY-1425 in subsets of AML and MDS
patients identified with the Company’s biomarker; the Company’s
strategies, plans and goals for SY-1425; and the potential benefits of
the Company’s gene control platform. The words ‘‘anticipate,’’
‘‘believe,’’ ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’
‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’
‘‘target,’’ ‘‘should,’’ ‘‘would,’’ and similar expressions are intended
to identify forward-looking statements, although not all forward-looking
statements contain these identifying words. Actual results or events
could differ materially from the plans, intentions and expectations
disclosed in these forward-looking statements as a result of various
important factors, including: Syros’ ability to: advance the development
of its programs, including SY-1425, under the timelines it projects in
current and future clinical trials; obtain and maintain patent
protection for its drug candidates and the freedom to operate under
third party intellectual property; demonstrate in any current and future
clinical trials the requisite safety, efficacy and combinability of its
drug candidates; obtain and maintain necessary regulatory approvals;
identify, enter into and maintain collaboration agreements with
third-parties; manage competition; manage expenses; raise the
substantial additional capital needed to achieve its business
objectives; and successfully execute on its business strategies; risks
described under the caption “Risk Factors” in the Company’s Quarterly
Report on Form 10-Q for the quarter ended June 30, 2016, which is on
file with the Securities and Exchange Commission; and risks described in
other filings that the Company makes with the Securities and Exchange
Commission in the future. Any forward-looking statements contained in
this press release speak only as of the date hereof, and the Company
expressly disclaims any obligation to update any forward-looking
statements, whether because of new information, future events or
otherwise.